Cell Therapy News/Articles, page-17329

“The proposal is the proposed trial in adults for aGVHD.It's not at all clear if the "proposed" single arm trial will be acceptable.” - whytee

Dachopper’s post #65408113 in Dec22 should answer your question indirectly about whether the FDA would look seriously at approving a single arm adult GVHD trial design, not to mention the fact that the recruits would be those who have failed Ruxolitinib, and the FDA’s own draft guidance as recent as Sep 2023 for industry about the treatment and prevention of GVHD, where it says single arm trials could be considered.

Dr Silviu Itescu - CEO Mesoblast Sep 30th webcast 2022" In particular the only drug that has been approved to date for SrGvHD is Ruxolitinib. It was approved on the basis of a single open arm study, when it subsequently did a randomised control trial it was required to do in a post approval commitment, there was no survival benefit in Sr GvHD. Independent investigators more recently published a significant survival benefit with remestemcel in children with the highest risk of mortality where survival in the controls was in the order of 10%, and survival in remestemcel treated children was in the order of 64%. “

Perhaps some reading comprehension lessons are in order?